Ignite Creation Date:
2025-12-24 @ 11:17 PM
Ignite Modification Date:
2025-12-25 @ 8:56 PM
Study NCT ID:
NCT06064656
Status:
COMPLETED
Last Update Posted:
2025-07-03
First Post:
2023-09-11
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009221', 'term': 'Myositis Ossificans'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009999', 'term': 'Ossification, Heterotopic'}, {'id': 'D009085', 'term': 'Mucopolysaccharidosis IV'}], 'ancestors': [{'id': 'D009220', 'term': 'Myositis'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 131}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2023-06-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-06', 'completionDateStruct': {'date': '2023-09-29', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-06-30', 'studyFirstSubmitDate': '2023-09-11', 'studyFirstSubmitQcDate': '2023-09-26', 'lastUpdatePostDateStruct': {'date': '2025-07-03', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-10-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-09-22', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Demographic characteristics', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of cardiovascular disease', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of metabolic disease', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of hematology disease', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of infection', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of respiratory disease', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of reproductive disease', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of neurologic disorders', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of skeletal disorders', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of gastrointestinal disease', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of hearing loss', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of dermatologic disorders', 'timeFrame': 'Up to 5 years'}, {'measure': 'Prevalence of neoplasms', 'timeFrame': 'Up to 5 years'}, {'measure': 'Average number of dispensations per FOP patient per year', 'timeFrame': 'Up to 5 years'}, {'measure': 'Cumulative number of dispensations per FOP patient', 'timeFrame': 'Up to 5 years'}, {'measure': 'Incidence of all-cause death', 'timeFrame': 'Up to 5 years', 'description': 'FOP Sub-Cohorts 1 and 2 Only'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Genetic disease', 'Heterotopic ossification (HO)', 'Skeletal dysplasia', 'Activin A receptor, type I (ACVR1)'], 'conditions': ['Fibrodysplasia Ossificans Progressiva (FOP)']}, 'descriptionModule': {'briefSummary': 'Primary Objective(s):\n\n* To describe the demographic characteristics of people living with FOP and a matched cohort of non-FOP people living in the United States (U.S.)\n* To describe the prevalence of clinical characteristics of interest and the use of key medications in people living with FOP and a matched cohort of non-FOP people living in the U.S.\n* To compare the prevalence of key clinical characteristics and medication use in people living with FOP to a matched cohort of non-FOP people living in the U.S.\n* To estimate the crude mortality rate among people living with FOP'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'FOP Cohort(s):\n\nThe International Fibrodysplasia Ossificans Progressiva Association (IFOPA) membership has 235 confirmed people living with FOP in the US. From this membership database, there were 131 members or next of kin of members who opted-in to allow for the tokenization of their membership data to occur.\n\nComparator Cohort:\n\nA random sample of 100,000 people from an insured population without FOP diagnosis living in the U.S. and matched to the FOP population.', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\nFOP Cohort(s):\n\n1. Living with FOP in the United States who have been identified from the IFOPA membership database\n2. Confirmed FOP diagnosis through IFOPA membership database will be tokenized and linked with HealthVerity's medical claims data, pharmacy claims data, laboratory data and obituary data, as defined in the protocol\n\nComparator Cohort:\n\n1. People without FOP living in the U.S. randomly sampled from HealthVerity's closed medical claims data and matched to the FOP population\n2. Index date are defined as each person's cohort entry date. People may exit and re-enter the cohort, with the period during which they were not in the cohort excluded from the analysis\n3. The comparator cohort will be matched to the cohort of people with FOP on the following variables: age, sex, insurance type and first index date\n\nExclusion Criteria:\n\n1\\. Failure to meet inclusion criteria"}, 'identificationModule': {'nctId': 'NCT06064656', 'briefTitle': 'A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Regeneron Pharmaceuticals'}, 'officialTitle': 'Clinical Characteristics and Mortality in Patients With Fibrodysplasia Ossificans Progressiva (FOP): A Non-Interventional US National Retrospective Cohort', 'orgStudyIdInfo': {'id': 'R2477-FOP-2143'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'FOP Cohort', 'description': 'Sub-Cohort 1: All patients with active enrollment (closed claims) during part or all the study period (2018-2023) Sub-Cohort 2: All patients with both active enrollment (closed claims) and open claims encounters during study period (2018-2022)', 'interventionNames': ['Other: Non-Interventional']}, {'label': 'Comparator Cohort', 'description': 'All individuals without FOP and with active enrollment (closed claims) during part or all of the study period (2018-2023)', 'interventionNames': ['Other: Non-Interventional']}], 'interventions': [{'name': 'Non-Interventional', 'type': 'OTHER', 'description': 'No study treatment will be administered in this study.', 'armGroupLabels': ['Comparator Cohort', 'FOP Cohort']}]}, 'contactsLocationsModule': {'locations': [{'zip': '10591', 'city': 'Tarrytown', 'state': 'New York', 'country': 'United States', 'facility': 'Regeneron', 'geoPoint': {'lat': 41.07621, 'lon': -73.85875}}], 'overallOfficials': [{'name': 'Clinical Trial Management', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Regeneron Pharmaceuticals'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Regeneron Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}